Nonclinical

The nonclinical stage of drug development serves as a critical bridge between initial compound discovery and the initiation of testing in humans.

During this phase, researchers generate a comprehensive body of nonclinical evidence to characterize a drug candidate’s pharmacological activity, pharmacokinetic (PK) and pharmacodynamic (PD) properties, and potential safety concerns through in silico, in vitro, and animal studies.

The results of nonclinical research are essential for supporting an initial application to regulatory authorities to perform a new clinical trial. Regulators rely on these data to evaluate potential risks, establish starting doses, define safety-monitoring strategies, and determine whether proposed clinical studies are ethically and scientifically justified.

Nonclinical programmes focus on supporting first-in-human studies (often in healthy participants), with limited early consideration of populations with distinct physiological characteristics, including pregnant and lactating women. After the first-in-human studies are initiated, additional nonclinical studies, including development and reproductive toxicology studies (DART) studies, provide information on the potential risks to pregnant and lactating women, their fetuses, and breastfed infants to inform safety monitoring and/or restrictions for clinical research in these populations.

For drugs intended for use during pregnancy or lactation – or where inadvertent exposure is likely – nonclinical research takes on heightened importance. Earlier and thoughtful integration of nonclinical DART studies may enable more informed decision-making and allow inclusion of these populations in later clinical trials. Including these populations in clinical trials reduces reliance on post-marketing evidence alone, and supports clearer risk–benefit assessments for clinicians and patients.